NYSCF News Update | Vol 10, Issue 2

NEWSupdate

N YS C F

VOLUME 10: ISSUE 2: 2017

The New York Stem Cell Foundation Research Institute

PAT I E N T S : K E Y T O N E W A L Z H E I M E R ’ S R E S E A R C H Three sisters, two with a genetic mutation, enable cutting-edge research and possible cell replacement therapies for neurological disorders.

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lzheimer’s is a devastating disease. It’s also a hard disease to study. By the time patients are diagnosed, a lot of the damage has already been done, masking what is going wrong at the beginning of the disease process. That’s where a recent study from NYSCF investigators and collaborators at the Icahn School of Medicine at Mount Sinai comes in.

The researchers successfully refined a technique to turn skin cells from patients with mild cognitive impairment (the beginning sign of Alzheimer’s) into the brain cells that degenerate first in Alzheimer’s disease. By recreating these cells, called basal forebrain cholinergic neurons, we can study how they behave in the early stages of the disease and look for ways to correct their dysfunction.

This research was made possible by the participation of three sisters: two with a gene that causes early-onset familial Alzheimer’s disease, and one without the gene. The researchers collected skin cells from each sister and then reprogrammed them to become basal forebrain cholinergic neurons. With an Alzheimer’s model that shows us

Neurons generated from an Alzheimer’s patient’s stem cells Image by Yosif Ganat, PhD, The NYSCF Research Institute

where things start to go wrong, we can start looking for ways to tackle the neuron dysfunction before it takes over.

NYSCF researchers then took the cell samples from the two sisters who carry the Alzheimer’s gene and corrected their genetic mutation using a gene editing technique called CRISPR-Cas9. When they generated neurons from the corrected cells, the neurons developed normally.

This research suggests that a cell replacement therapy for Alzheimer’s based on these steps might be possible, giving us a way to replace a patient’s degenerating cells with healthy ones created in the lab.

Future studies will test this approach in animal models before moving on to clinical trials in patients.

Scientists at the NYSCF Research Institute

Alzheimer’s disease affects five million people in the United States today and is projected to affect sixteen million people by 2050. It is a problem in urgent need of solutions, and one that NYSCF is committed to eradicating. This research is an important step toward understanding Alzheimer’s and developing effective new therapies to treat and even cure it.

F E AT U R E D I N T H I S I S S U E G A L A & SCIENCE FA IR

S T E M CE L L PR I Z E

CONFER ENCE

CON V ENI NG

NYSCF Honors Stem Cell Heroes at Annual Gala p.3

Awarded to Dr. Paul J. Tesar for his pioneering regenerative medicine discoveries p.4

Translational research, collaboration, and advocacy p.6

Leaders in stem cell science meet to share their new ideas here at NYSCF p.7

Contact us at info@nyscf.org or 212.787.4111

W W W. N Y S C F. O R G

U N I T I N G T O A C C E L E R AT E C U R E S BOARD OF DIRECTORS Roy Geronemus, MD, Chairman Susan L. Solomon, CEO Margo Alexander Peggy Brim Bewkes Marilyn G. Breslow Karen E. Burke, MD, PhD Paul Goldberger George Lazarus, MD Richard J. Massey, PhD Stephen M. Ross Stephen M. Scherr Stuart Smith, DPhil Anita Volz Wien

LEADERSHIP COUNCIL Katherine Bristor & William Priest David A. Carmel Russell L. Carson Chuck Close Alan M. Cohen Shirley Cook Fiona Druckenmiller Jodie & John Eastman Frank Gehry Lawrence E. Golub & Karen Finerman April Gornik Marlene Hess Tania Higgins Dorothy Lichtenstein Sandra Lloyd Paul M. Meister Stephen Meringoff David Mitnick Nancy & Fred Poses Carol Roaman Julian Robertson Andy Russell Barbara Stovall Smith Noelle & Dick Wolf

JUNIOR LEADERSHIP COUNCIL (EXECUTIVE BOARD) Sabrina Bertucci Daniel Brancusi Francesco Clark Maggie Close Carolyna De Laurentiis Alex Goldberger, Co-Chair Daniyal Hussain Brandon Law Mark McCauley, Co-Chair Charlotte Meringoff Craig J. Moskowitz, MD Max Mullen Neil O’Donnell, Esq. Thomas J. Pacchia Melissa Rothberg Richard W. Rundle Ben Sherman

NYSCFNEWSupdate

Dear Friends, This year has been one of milestones for NYSCF. We moved our global headquarters to a new home in Midtown Manhattan. Our state-of-theart facility includes a laboratory equipped with cutting-edge technologies as well as a lecture space where leaders in the field are sharing their work with others dedicated to accelerating cures for major diseases.

NYSCF is accelerating cures and treatments through collaboration throughout the entire regenerative medicine community.

This year’s Tokyo – New York Stem Cell Summit brought researchers from NYSCF and the University of Tokyo together to share the latest research advancements and best practices. NYSCF and the Chan-Zuckerberg Initiative hosted a two-day workshop where scientists exchanged ideas and mapped out critical needs and next steps for the field of stem cell research. And The NYSCF Conference, held this October at The Rockefeller University, featured presentations on groundbreaking translational research from stem cell leaders around the world.

Translational research – moving effective treatments out of the lab and into the clinic – is our ultimate goal. The NYSCF Research Institute is currently pursuing that goal with two pioneering programs: a physician-sponsored clinical trial for macular degeneration with physicians at Columbia University Medical Center and a partnership with Hospital for Special Surgery to explore stem cell-based solutions to orthopedic injuries. We hope these programs will offer relief to patients by applying personalized medicine to real-life cases of disease and injury.

Thank you all for your ongoing and generous support. None of our exciting advances would be possible without private philanthropy. With your help, we are getting closer each day to finding lifesaving cures through stem cell research.

Warmest wishes for a happy and healthy New Year.

Susan L. Solomon CEO and Co-Founder

NYSCF PERSONALIZED MEDICINE SERIES

Turning Research into Cures Stay tuned for upcoming talks at

N Y S C F. O R G / S E R I E S 2

THE NYSCF GAL A AND SCIENCE FAIR

N YSCF proudly honored three Stem Cell Heroes at the 2017 NYSCF Gala and Science Fair, recognizing Andreas C. Dracopoulos with the NYSCF Humanitarian Award, NYSCF Board of Directors Chairman Roy Geronemus, MD, with the NYSCF Leadership Award, and Clifford Ross with the inaugural NYSCF Arts Leadership Award.

Top row from left to right: Andreas C. Dracopoulos, Susan L. Solomon, Clifford Ross; Stephen M. Scherr, Roy Geronemus, MD; Middle row from left to right: Dorothy Lichtenstein,Toni Ross; Anita Volz Wien, Scott Noggle, PhD; Marlene Hess, Peter Nadosy; David Walentas, Jane Walentas; Bottom row from left to right: Bruce Warwick, Margie Warwick; Edward Bleier, Laura Landro, Rick Salomon; Ian Schrager, Maya Lin. 3

NYSCFNEWSupdate

PAU L TE SA R AWA R D E D TH E N YS C F – RO B E RT SO N STE M C E L L PR I Z E

N E W I NVE STIGATOR C L A S S

N YSCF welcomed six top scientists into the 2017 class of NYSCF – Robertson Investigators. These leading researchers join the elite global NYSCF Innovator Community of over 150 scientists, including those at the NYSCF Research Institute in New York.

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he 2017 NYSCF – Robertson Stem Cell Prize recognized Dr. Paul J. Tesar for his pathbreaking, translational research.

An Associate Professor at Case Western Reserve University and a NYSCF – Robertson Stem Cell Investigator Alumnus, Dr. Tesar has accelerated the field of regenerative medicine through his discovery of pluripotent epiblast stem cells and has worked to develop technologies that utilize stem cells to treat multiple sclerosis and other devastating neurological disorders.

N Y S C F – RO B E RT S O N S T E M C E L L I N V E S T I G ATO R S

Sergiu Pasca, MD

Stanford University Dr. Pasca develops new approaches for studying human brain development, including building mini brain organoids, and for uncovering the mechanisms of neuropsychiatric disorders.

Louis Vermeulen, MD, PhD

Academic Medical Center, Amsterdam, The Netherlands Dr. Vermeulen focuses on the role stem cells play in colorectal cancer development and progression with an aim to improve prevention and treatment strategies.

Brian Wainger, MD, PhD

Left: Paul J. Tesar, DPhil, with the NYSCF – Robertson Stem Cell Prize

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Massachusetts General Hospital Dr. Wainger develops precision medicine platforms using stem cells to build more powerful human models in the laboratory.

He is also conducting clinical trials in patients with amyotrophic lateral sclerosis (ALS).

Right: Myelinated axons

Image by Valentina Fossati, PhD, The NYSCF Research Institute

This prize recognizes the importance of stem cell research and will accelerate our efforts to rapidly and safely bring new stem cell-based therapies to patients.

N Y S C F – RO B E RT S O N N E U RO S C I E N C E I N V E S T I G ATO R S

Stephen Brohawn, PhD

University of California, Berkeley Dr. Brohawn studies how the nervous system senses and responds to physical forces, including hearing, touch, balance, pain, breathing, digestion, and blood pressure control.

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The Tesar laboratory has recently focused on studying myelin – the insulating coating on nerve cells that helps them send signals – and the diseases that impact it, developing new techniques for producing myelinating cell types. Cells developed by the Tesar laboratory have been used to screen new drug candidates, enable advances in stem cell biology, and translate this work into clinical use.

Gregory Scherrer, PhD, PharmD

Stanford University School of Medicine Dr. Scherrer researches how to alter the brain’s interpretation of pain signals to eliminate the unpleasantness associated with pain and restore patients’ quality of life.

“This recognition is truly an honor for me and my scientific team,” said Dr. Tesar. “This prize recognizes the importance of stem cell research and will accelerate our efforts to rapidly and safely bring new stem cell-based therapies to patients.”

Ilana Witten, PhD

Princeton University Dr. Witten studies the neural circuits for reward learning and decision-making with a focus on the role of dynamics and feedback to provide insights into how actions are linked to later occurring outcomes.

The NYSCF – Robertson Stem Cell Prize has been awarded annually since 2011 to an outstanding young stem cell scientist in recognition of significant and path breaking translational stem cell research.

NYSCFNEWSupdate

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A C C E L E R AT I N G C U R E S T H R O U G H C O L L A B O R AT I O N

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esearch works best when it’s collaborative. Sharing ideas, combining resources, and working together toward a common goal makes the process of studying disease and developing therapies more effective. Here are some of the amazing current collaborations between NYSCF Innovators.

TACKLING T YPE 1 DIABETES

DEVELOPING DRUGS FOR PTSD

Bjarki Johannesson, PhD

Kristen Brennand, PhD

NYSCF – Robertson Stem Cell Investigator; Icahn School of Medicine at Mount Sinai

Investigator, The NYSCF Research Institute

& Deepta Bhattacharya, PhD

& Scott Noggle, PhD

NYSCF – Robertson Stem Cell Investigator, University of Arizona

Senior Vice President, Research, The NYSCF Research Institute

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D rs. Johannesson and Bhattacharya are working on a therapy for type 1 diabetes, which results from an immune

TSD affects those who have undergone a traumatic event, often leaving patients with flashbacks, anxiety, and chronic pain. Testing and developing drugs for PTSD has long been a challenge for researchers. Drs. Brennand and Noggle are using stem cells from veterans to screen hundreds of existing and novel drugs, shedding light on what works to treat this disorder and identifying strategies for developing new treatments.

attack on insulin-producing cells in the pancreas. By generating insulin-producing cells from embryonic stem cells, Drs. Johannesson and Bhattacharya hope to create an effective cell replacement therapy for the millions of people suffering from this disease.

N Y S C F I N N O VAT O R B R E A K T H R O U G H : First Ever System to Mass-Produce Bioengineered Livers

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YSCF – Robertson Stem Cell Investigator Takanori Takebe, MD, of Cincinnati Children’s Hospital Medical Center and Yokohama City University in Japan, led a study demonstrating the first successful system for mass produced, bioengineered livers.

Published in Cell Reports, the researchers created three different derived cell types from induced pluripotent stem (iPS) cells, then combined the three cell types in specially engineered plates to engineer so-called “liver micro-buds.” This process yielded tens of thousands of these liver micro-buds per batch, resulting in functionally significant quantities for use in cell replacement therapies and toxicity testing.

Takanori Takebe, MD

To test the therapeutic proof of principle, the researchers challenged the functionality of the equivalent of one batch of liver micro-buds in mouse models of liver disease. In an exciting result, the micro-buds rescued the mice from liver disease. Significantly, the process to create liver micro-buds uses only iPS cells and no animal feeder byproducts, typically used to make cells for research purposes, smoothing the path for eventual use in humans.

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NYSCFNEWSupdate

THE NYSCF CONFERENCE: Translational Research, Collaboration, and Advocacy

“A t first,

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veryone in Caspary Auditorium at The Rockefeller University is looking at an eye chart, but they aren’t taking an eye test. They’re being shown what Elizabeth – a patient with age-related macular degeneration (AMD) – sees.

the only letter

Elizabeth could see was the top letter...

This is how well she can see now ”

Lund University’s Malin Parmar, PhD, is a NYSCF – Robertson Investigator who, like Dr. Coffey, is also conducting groundbreaking translational research. Dr. Parmar focuses on Parkinson’s disease – specifically, how neurons derived from a patient’s own cells can be used to replace the neurons that the disease attacks.

Pete Coffey, DPhil

“With Parkinson’s, we know that the problem comes from death of a specific group of cells, so using a cell replacement therapy to treat it makes a lot of sense,” explains Dr. Parmar, who is dedicated to helping the millions of people affected by the disease.

“At first, the only letter Elizabeth could see was the top letter,” says University College London and University of California, Santa Barbara’s Pete Coffey, DPhil, as he points to the giant “A” that has been circled at the top of a pyramid of letters. Then the eye chart is shown again, this time with six much smaller letters circled toward the bottom. “This is how well she can see now,” explains Dr. Coffey.

AMD causes degeneration of eye cells and loss of vision. Dr. Coffey and his collaborators found a way to implant a patch containing non-diseased eye cells made from stem cells into the eye to replace the degenerating ones. This is the treatment Elizabeth received, and it’s why her vision has improved so much.

Dr. Coffey’s presentation is just one example of the many captivating talks at the 2017 NYSCF Conference in October. The conference served as a forum for leaders in translational stem cell research to discuss their work and share ideas for the future of the field.

Actress, screenwriter, and activist Lauren Miller Rogen gives a patient advocate keynote at The NYSCF Conference

Stem cell leaders and pioneers, including Harvard Stem Cell Institute’s Douglas A. Melton, PhD, and Gordon Keller, PhD, from the McEwen Center for Regenerative Medicine, discussed their latest work using stem cell technology to research and develop treatments for diabetes and heart disease in a disease-focused session.

Stanford University’s Irving Weissman, MD, one of the founders of the stem cell research field, joined SEngine CEO Carla Grandori, MD, PhD, Memorial Sloan Kettering Cancer Center Physician-inChief and leading cancer expert José Baselga, MD, PhD, journalist Laura Landro, and NYSCF CEO Susan L. Solomon, JD, to discuss the latest regenerative medicine advancements in cancer treatment.

Left to right: Susan L. Solomon, JD, Ir ving Weissman, MD, Carla Grandori, MD, PhD, José Baselga, MD, PhD, and Laura Landro

NYSCFNEWSupdate

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CONVENING THE STEM CELL COMMUNITY

One of the cornerstones of advancement is the sharing of success stories and new ideas. By hosting and participating in meetings of stem cell leaders, NYSCF is helping to ensure that groundbreaking research is discussed outside the lab.

C HAN -ZUC K E RB E RG I N ITIATIVE AN D NYSC F CO - HOST COLL ABOR ATIVE WORKS HOP

N YSCF recently opened its doors to the regenerative medicine community for a meeting co-hosted by the Chan-Zuckerberg

Initiative. This initiative, founded by Facebook’s Mark Zuckerberg and his wife, Priscilla Chan in 2015, supports promising biomedical research by investing in bold ideas and large-scale stem cell efforts.

The meeting allowed attendees to share their own bold ideas and discuss health and science research with others committed to making a difference in the field. By including presentations as well as discussion and debate, attendees leveraged the opportunity to both speak about their own advancements and work together to plan for the future.

Susan L. Solomon, JD, and fellow stem cell leaders share ideas at a meeting co-hosted with the Chan-Zuckerberg Initiative at NYSCF headquar ters.

G L O B A L R E S E A R C H TA K E S C E N T E R S TA G E AT T O K YO – N E W YO R K S T E M CELL SUMMIT

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esearchers from the University of Tokyo and NYSCF met to discuss recent research developments and how to enhance the field of stem cell research globally.

Keynotes from Kevin Eggan, PhD, Principal Investigator at the Harvard Stem Cell Institute, and Hiromitsu Nakauchi, MD, PhD, Professor of Genetics at Stanford University, focused on translational advancements in stem cell research, and many other researchers from both organizations shared their lab’s groundbreaking findings.

Collaboration and discussion between researchers from around the world ensures that new ideas and perspectives are being shared, leading to a more comprehensive understanding of the field amongst its leaders.

University of Tokyo researchers Hiromitsu Nakauchi, MD, PhD, and Shoji Takeuchi, PhD, and NYSCF Investigators Valentina Fossati, PhD, and Scott Noggle, PhD, engage in a panel discussion on stem cell organoids.

NYSCF PERSONALIZED MEDICINE SERIES

T H E G E N E E D I T I N G T E C H N O LO G Y R E VO LU T I O N I Z I N G R E S E A RC H

N YSCF kicked off the 2017-2018 Personized Medicine Series with a talk from CRISPRCas9 inventor, MIT Professor, and NYSCF – Robertson Investigator Feng Zhang, PhD.

Dr. Zhang spoke about CRISPR, the revolutionary gene editing tool he helped pioneer, with the NYSCF community at NYSCF headquarters in New York.

Dr. Zhang explained that CRISPR works a bit like Microsoft Word. In Microsoft Word, you can search for certain words in a document and then modify, delete, or replace them.

CRISPR does this for genes, allowing scientists to locate and edit faulty stretches of DNA within our genome. It can be used to study or treat pretty much any disease with a genetic component – cancer, Huntington’s, Alzheimer’s, etc. Dr. Zhang emphasized the importance of this technology for the future of research and engaged in a Q&A about its applications.

Feng Zhang, PhD

Join us for future talks. Learn more at N Y S C F . O R G / S E R I E S 7

NYSCFNEWSupdate

FUTURE CURES B E G I N W I T H YO U R S U P P O R T T O D AY !

Please make a gift to help NYSCF advance stem cell research toward the clinic.

You can donate online at www.nyscf.org/donate or mail a check to:

619 W 54th Street New York, NY 10019

THE NEW YORK STEM CELL FOUNDATION 619 WEST 54 TH STREET, NEW YORK, NY 10019 FACEBOOK.COM/NYSCF TWITTER.COM/NYSCF INSTAGRAM.COM/NYSCF VIMEO.COM/NYSCF

NYSCFNEWSupdate N Y S C F N EWS U PDAT E

The NYSCF Gala and Science Fair NYSCFNEWSupdate